BioMarin files BLA for Vimizim in USA

US drugmaker BioMarin Pharmaceutical (Nasdaq: BMRN) has submitted a Biologics License Application (BLA) to the Food and Drug Administration for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. The company also intends to submit an application for registration in the European Union by the end of April 2013.

"Based on the positive results from our Phase III pivotal study, we believe that Vimizim offers a substantial benefit to patients with MPS IVA, a severely debilitating and progressive disease for which there is no current treatment," said Hank Fuchs, chief medical officer of BioMarin.

"The submission of the BLA represents a significant milestone for BioMarin and is the result of the strong, collaborative effort of many hard working employees, investigators, patients, and their families. With this application, BioMarin continues in its long-standing tradition of developing important therapies for those who are most in need. We look forward to working with the US regulatory authorities to bring this treatment to patients," Dr Fuchs added.